Agios Pharmaceuticals, Inc.

Good morning and welcome to the IGL's second quarter 2023 conference call. At this time, all participants are in the listen-only mode. There will be a question and answer session at the end. Please be advised that this call is being recorded at IGL's request. I would now like to turn the call over to Chris Taylor, Vice President, Investor Relations and Corporate Communications for IGL's.

Thank you, Operator. Good morning, everyone, and welcome to IGL's second quarter 2023 conference call. You can access slides for today's call by going to the Investors section of our website, agios.com. On today's call, I am joined by our Chief Executive Officer, Brian Goff, Dr. Sarah Hewins, Chief Medical Officer and Head of Research and Development, Sveta Milanova, our Chief Commercial Officer, and Cecilia Jones, Chief Financial Officer. Before we get started, I would like to remind everyone that some of the statements we make on this call will include forward-looking statements. Actual events and results could differ materially from those expressed or implied by any forward-looking statements as a result of risks, uncertainties, and other factors, including those set forth in our most recent filings with the SEC and any other future filings that we may make with the SEC. And with that, I'll turn the call over to Brian.

Thanks, Chris. Good morning, everyone, and thank you for joining us. Augiosis focused on delivering transformative therapies for patients living with rare diseases, and in particular, we're the pioneering leader in PK activation focused on hematologic diseases. In the second quarter, we made significant progress advancing our industry-leading pipeline of PK activators targeting hematologic diseases that share a common underlying pathophysiology. With each step forward, each data readout, The probability of success for the platform is strengthened, and we are excited to share our updates with you today. As we articulated at the beginning of this year, we're prioritizing potential business development opportunities based on five key criteria. Rare disease focus, transformative for patients, an identified regulatory pathway, potential to de-risk early, and a clear path to value creation. Earlier this morning, we were very pleased to announce a license agreement with Alnylam Pharmaceuticals, the leading RNAi therapeutics company that is highly aligned with these five criteria. Under this agreement, Agios will acquire the rights to develop and commercialize Alnylam's novel preclinical siRNA for the potential treatment of polycythemia vira, or PV. PV is a rare and potentially fatal hematologic disease that affects approximately 100,000 patients in the US and for which phlebotomy is the standard of care. Our goal is to address the high unmet need in PV by delivering a convenient disease-modifying treatment option that reduces or eliminates the need for phlebotomy. This agreement is therefore aligned not only with our business development strategy, but also our core scientific expertise and clinical and commercial capabilities in rare hematology. We look forward to initiating IND-enabling studies later this year. Sarah will provide more detail on the siRNA development candidate in just a few minutes. Also this quarter, we announced positive results from the Phase II portion of the operationally seamless Phase II-III Rise-Up Study of Minitivet in Sickle Cell Disease. The study met the primary endpoint of hemoglobin response for patients in both mid to pivot treatment arms. And in recent weeks, our team has continued to analyze the results and has selected the 100 milligram dose for the phase three portion of the study. We are now focused on phase three execution and are quite eager to enroll the first patient later this year. Broadly, these results add to the growing body of consistent and compelling data that we have continued to generate with our PK activators, highlighting the potential of this differentiated mechanism of action to transform patient function, quality of life, and long-term outcomes across multiple disease areas. In fact, with more than eight years of clinical experience and the largest data set for any PK activator, Pyrakyne has demonstrated consistent results across three distinct diseases. In this context, we were also pleased to announce this quarter that we've completed enrollment in both Phase III studies of midipivet in thalassemia, as well as the Phase IIa study of our novel PK activator, AG946, in lower-risk MDFs. This progress reflects our operational excellence in clinical development and investigators' enthusiasm for the potential of PK activation in these indications. Based on this progress, we continue to expect two readouts from the ENERGYZE and ENERGYZE-T Phase III studies in thalassemia next year, and we've pulled forward the expected timing of the top-line results for the Phase IIa study in lower-risk MDS to the end of this year. Turning to our commercial business, we're encouraged to see that the consistent and compelling efficacy of Mitipivet observed in the clinical trial experience has continued to translate to persistency on therapy among adults living with PK deficiency in the real world. As we continue to maximize the opportunity in the current launch in PK deficiency, we're building the capabilities needed to fully realize the potential of anticipated future launches in thalassemia, sickle cell disease, and lower-risk MDS. Sveta will provide a detailed update on our commercial performance in just a few minutes. As you'll hear from Cecilia, we ended the second quarter with a cash position of nearly $950 million on the balance sheet. One brief reminder, as part of the divestiture of our oncology business to survey in 2021, we retain the rights to a potential $200 million milestone upon FDA approval of voracidinib and royalties on potential U.S. net sales. We were encouraged by the results of Servier's Phase III trial, and we look forward to tracking next steps. We're expecting a number of additional milestones by the end of the year, including enrolling more than half of the patients in the Phase III ACTIVATE Kids and ACTIVATE Kids T studies of midipivac in pediatric PK deficiency, filing the IND for our pH stabilizer for the treatment of PKU, and the newly added milestone the data readout from the Phase 2A study of AG946 in lower-risk MDS. We're very enthusiastic about the clinical development momentum we're building and look forward to anticipated readouts from the Phase 3 studies of midipivette and thalassemia in 2024 and readouts from the Phase 3 studies of midipivette in sickle cell disease and pediatric PK deficiency in 2025. With that, I'll now turn the call over to Sarah.